33 TWENTYFOURSEVENBIOPHARMA Issue 2 / June 2025 going to be a challenge. …we’ve got good people, and I’m not worried about their basic understanding. “…we’re doing better than most people imagine, but the field is changing exponentially, not linearly. So it is a worry. And as generative AI comes in and the whole space of consumer interactions with devices coming into play in the nonmedical area,…” Although less visible, a range of resources were also allocated to the FDA to support these faster review timelines and cultivate a more dynamic regulatory environment. The increase in funding gave the agency wider scope to expand its workforce and refine its review processes, supporting the accelerated approval of effective therapies. The impact of shifting policies By refining approval pathways and embracing realworld evidence, the FDA has been able to authorise the market entry of promising new therapies. Indeed, pharmaceutical firms have been able to make these drugs available much sooner to those most in need, where time is of the essence—such as oncology and rare genetic disorders. Regarding their investigational gene therapy, isaralgagene civaparvovec (ST-920), Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics, stated: “I strongly believe in the potential for ST-920 to alleviate many manifestations of Fabry disease and am delighted to have a clear regulatory pathway that could bring this treatment to patients significantly sooner than originally anticipated.” Other drug bosses, such as the CEO of Israel’s Teva Pharmaceutical Industries, have demanded more saying: “In the FDA (Food and Drug Administration), there is no speed.” “I would like them to do things on a regular time because the more generics we bring, the more savings we bring to the United States healthcare system, so just approve them,” Richard Francis told Reuters. “Many of these products are approved in Europe.” He added that drugs, such as Huntington’s treatment Austedo, should be exempt from funding restrictions. Trial designs and endpoints But where the updated guidance has been most felt has been its impact on adaptive trial designs and acceptance of novel endpoints that have allowed companies to conduct more efficient clinical studies. This flexibility has reduced both the cost and duration of clinical trials while improving data quality. Regulators now have a more nuanced understanding of a drug’s safety and efficacy, which in turn supports more informed decision-making. This streamlining is certainly needed. As drugs enter each clinical trial stage, the spiralling costs would make even the biggest pharma corporations break out in a cold sweat. According to The Gates Foundation, a vaccine for tuberculosis, in which a smaller share of the population is infected during the study period, can cost up to $550m (€508m) for a phase 3 trial. That is at the extreme end. A paper looking into vaccines for rotavirus reported an estimate of $2,000 to $3,000 per patient for phase 3 trials alone, whilst research published in The Lancet suggests that the 5,500-person trial of the R21 malaria vaccine could have cost around $11m (€10.1m). The same could be said about novel endpoints such as surrogate markers, which also speed up the approval of new therapies. The FDA’s AAP currently uses surrogate endpoints to approve drugs for serious conditions, possibly cutting the time to market. However, this approach has raised questions about the strength of evidence about the drugs’ effectiveness and the need for confirmatory trials post-approval. Industry reaction The impact of these reforms is already being felt with drug approvals moving faster and digital health innovations experiencing a wave of regulatory backing. “By permitting earlier access to novel therapies, the accelerated approval pathway has helped extend, and even save patients’ lives,” said Eli Lilly and Company of AAP’s benefits. However, some argue that adaptive trial designs and novel endpoints have introduced efficiencies that benefit both patients and drug developers. Others REGULATION
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